Duke-Margolis & FDA: Workshop on stats & methods for rare diseases

Dear All:

Duke-Margolis and FDA have now announced a 19 Mar 2018 workshop entitled Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Drug Development (FR notice). Here’s the teaser from the web: “Convened by the Duke-Robert J. Margolis, MD, Center for Health Policy at Duke University and supported by a cooperative agreement with FDA, this public event will bring the stakeholder community together to discuss the challenges that rare disease settings pose for treatment development and regulatory decision-making, as well as the promising study designs and analytical methods that can help overcome them. The day’s discussion will center around novel approaches to trial design and analysis to improve efficiency and interpretability of results in rare disease trials.”

Note how neatly this fits with their previously announced 16 April 2018 1-day workshop on inclusion and exclusion criteria in clinical trials (corresponding FR notice): “The purpose of the public meeting is to bring the stakeholder community together to discuss a variety of topics related to eligibility criteria in clinical trials and their potential impact on patient access to investigational drugs, and how to facilitate the enrollment of a diverse patient population.” 

This is great to see! While neither of these two is specifically focused on AMR-related issues, the applicability to our trial design issues should be obvious. Unfortunately, I am probably going to miss both workshops but I hope that at least a few of you will get there, especially for the 19 Mar workshop on statistical design. If you do go, I’d love to share what you learned with the newsletter community … please let me know!

All best wishes, –jr

John H. Rex, MD | Chief Medical Officer, F2G Ltd. | Expert-in-Residence, Wellcome Trust. Follow me on Twitter: @JohnRex_NewAbx. See past newsletters and subscribe for the future: https://13.43.35.2/blog/

Upcoming meetings of interest to the AMR community:


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